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As gene therapies advance through clinical trials, more and more of these treatments are receiving approval from the U.S. Food and Drug Administration (FDA). Boston Children's Hospital now offers several FDA-approved gene therapy treatments, and we are actively participating in clinical trials in hopes of bringing additional approved gene therapies to our patients.

Current gene therapies we are offering include:

Beta thalassemia gene therapy (Zynteglo®)

Caused by mutations in the beta-globin gene, beta thalassemia prevents people from producing enough beta-globin, a key hemoglobin component. Boston Children’s Hospital is now offering Zynteglo®, also called beti-cel, to eligible children and adults with beta thalassemia who require regular transfusions of red blood cells. Zynteglo®, approved by the FDA in August 2022, delivers a functioning copy of the beta-globin gene into patients’ blood stem cells. Most patients who received the treatment have no longer needed blood transfusions.

CAR T-cell therapy (KYMRIAH™) for leukemia and lymphoma

CAR (chimeric antigen receptor) T-cell therapy is a promising treatment that alters the body's T-cells to better detect and destroy infected ‭or cancerous cells.‬‬‬‬‬‬

The Dana-Farber/Boston Children's Cancer and Blood Disorders Center is offering the first FDA-approved CAR T-cell therapy, called KYMRIAH™, to two groups of eligible patients:

Girl wearing bicycle helmet while standing on bike path

CAR T-cell therapy helps hockey fan ice her leukemia

Ava, a rising sixth grader, was one of the first patients to undergo CAR T-cell therapy for acute lymphoblastic leukemia (ALL) at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.

Retinal gene therapy (Luxturna™)

The Department of Ophthalmology at Boston Children’s Hospital is a Certified Center of Excellence for Luxturna™, an FDA-approved gene therapy for the treatment of inherited retinal eye disease in patients over 12 months of age with mutations in the RPE56 gene.

Cerebral adrenoleukodystrophy gene therapy (SKYSONA®)

Cerebral adrenoleukodystrophy (CALD) is a rare genetic condition that causes the buildup of very long chain fatty acids in the brain. Boston Children’s Hospital is now offering SKYSONA®, also called elivaldogene autotemce or eli-cel, to eligible boys with CALD who are not yet experiencing symptoms. Boston Children’s helped pioneer SKYSONA®, which was approved by the FDA in September 2022. It is designed to replace the defective or missing ABCD1 gene, which controls the production of an enzyme that our body normally uses to breaks down fatty acids.

Spinal muscular atrophy (SMA) gene therapy (Zolgensma®)

Boston Children’s Hospital is one of the first pediatric hospitals in the nation to offer Zolgensma®, an FDA-approved gene therapy for the treatment of spinal muscular atrophy (SMA) in children less than 2 years of age.

Learn about SMA gene therapy.

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Sofie’s story: A new gene therapy treatment for SMA

One year after receiving gene therapy treatment through a clinical trial at Boston Children’s Hospital, Sofie is not only able to breathe on her own and eat by mouth, but is also sitting up, moving her head, raising her hands, saying words, and rolling in her small wheelchair.